A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
Status: Active_not_recruiting
Location: See all (51) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 4
Maximum Age: 21
Healthy Volunteers: f
View:
• Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
• Ambulant or Non-Ambulant
• Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial including nusinersen and/or risdiplam and/or a history of onasemnogene abeparvovec
Locations
United States
Arizona
Phoenix Children's
Phoenix
California
UCSD & Rady Children's
La Jolla
Children's Hospital of Los Angeles
Los Angeles
UCSF Benioff Children's Hospital, Medical Center
San Francisco
Colorado
Children's Hospital Colorado
Aurora
Florida
UF Health, Shands Hospital
Gainesville
Georgia
Rare Disease Research
Atlanta
Iowa
University of Iowa
Iowa City
Illinois
Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Indiana
Indiana University -Riley Research
Indianapolis
Kansas
University of Kansas Medical Center
Fairway
Massachusetts
Boston Children's Hospital - Harvard
Boston
Michigan
BSHS Office of Research
Grand Rapids
Missouri
Washington University in St. Louis
St Louis
North Carolina
Duke University Medicine
Durham
New York
Columbia University Medical Center
New York
Stony Brook University Hospital
Stony Brook
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Nationwide Children's Hospital
Columbus
Pennsylvania
Penn State College of Medicine
Hershey
CHOP Children's Hospital of Philadelphia
Philadelphia
UPMC Children's Hospital of Pittsburgh
Pittsburgh
Tennessee
Vanderbilt University Medical Center
Nashville
Texas
UT Pediatric Neurosciences/Dell Children's Medical Center
Austin
Neurology Rare Disease Center
Denton
Cook Children's Hospital
Fort Worth
Virginia
University of Virginia Children's Hospital
Charlottesville
Children's Hospital of The King's Daughters
Norfolk
Washington
MultiCare Institute of Research and Innovation
Tacoma
Wisconsin
Medical College of Wisconsin
Milwaukee
Other Locations
Belgium
University Hospital Antwerp
Edegem
University Hospital Ghent
Ghent
University Hospital Leuven
Leuven
Germany
University Hospital Essen (Public-Law Institution) - Dept. of Pediatrics I
Essen
University Hospital Freiburg, Center For Children and Adolescent Medicine, Dept. of Neuropediatrics and Muscle Disorders
Freiburg Im Breisgau
Dr. Von Haunersches Children'S Hospital - Lmu Munich
Munich
Italy
Irccs Institute of Neurological Sciences of Bologna - Bellaria Hospital
Bologna
Nemo-Brescia Clinical Center For Neuromuscular Diseases
Gussago
IRCCS NEUROLOGICAL INSTITUTE C. MONDINO CHILD and NEUROPSYCHIATRIC UNIT
Pavia
Bambino Gesù Children'S Research Hospital Irccs - San Paolo Office Dept. of Neuroscience
Roma
Netherlands
University Medical Center Utrecht
Utrecht
Poland
University Clinical Centre in Gdansk - Dept. of Developmental Neurology
Gdansk
Heliodor Swiecicki Clinical Hospital At Medical University - Child and Adolescents Neurology Clinic
Poznan
The Children'S Memorial Health Institute - Dept. of Neurology and Epileptology
Warsaw
T. Marciniak Lower Silesian Specialist Hospital, Pediatric Neurology Dept.
Wroclaw
Spain
Donostia University Hospital
Donostia / San Sebastian
Hospital Sant Joan de Déu
Esplugues De Llobregat
Maternal-Child'S Hospital of Málaga, Regional University Hospital - Pediatric Neurology Unit
Málaga
La Fe University and Polytechnic Hospital
Valencia
United Kingdom
Royal Hospital For Children
Glasgow
John Radcliffe Hospital
Oxford
Time Frame
Start Date: 2022-07-06
Completion Date: 2026-01
Participants
Target number of participants: 269
Treatments
Experimental: taldefgrobep alfa
taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.~taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase.
Placebo_comparator: Placebo
Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase.~Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase.
Related Therapeutic Areas
Sponsors
Leads: Biohaven Pharmaceuticals, Inc.